US orphan status for AB Science's muscle-wasting drug

AB Science’s tyrosine kinase inhibitor masitinib has been awarded Orphan Drug status by the US Food and Drug Administration as a treatment for the rare degenerative disorder amyotrophic lateral sclerosis which causes progressive wasting and paralysis of voluntary muscles.

Around 15,000 people in the US are affected by the condition, with 4,500 new cases every year, and the prognosis for patients is extremely poor, with just 10% remaining alive five years post diagnosis.

According to the firm, because of its immunotherapy action masitinib can have an effect on survival, alone or in combination with chemotherapy.

The drug blocks mast cell-mediated degranulation, the release of cytotoxic substances that could further damage motor nerves, and is already marketed to treat certain cancers in dogs as Kinavet. Phase III trials in humans are currently underway.

Orphan drug status gives AB Science a seven-year period of marketing exclusivity for the drug in the US on approval, as well as the chance to apply for research grant funding for early-stage trials, tax credits for certain research expenses, and a waiver from the FDA's application user fee.